Evidence map›Paper›PMID 29430176›Full record

ReviewInternational journal of chronic obstructive pulmonary disease2018

Alpha 1 antitrypsin to treat lung disease in alpha 1 antitrypsin deficiency: recent developments and clinical implications.

Kenneth R Chapman, Joanna Chorostowska-Wynimko, A Rembert Koczulla, Ilaria Ferrarotti, Noel G McElvaney

Registry-linked trialOpen access · goldAbstract readReview
PubMed Full text at PMC Publisher
In one paragraph

Review in International journal of chronic obstructive pulmonary disease, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT06029543 (Permian Health Women's Lung Health Study), which is not on this map. Cited by 27 papers, 3 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
27citing papers in PubMed, 3 pooled it
2.0field-weighted citation impact, top 15% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

NCT06029543 not yet recruitingnot on this mapstarted 2023, after this paper: background citation

Permian Health Women's Lung Health Study

Typeobservational_patient_registrySponsorPermian Health Lung InstituteRan2023 to 2026Enrolled350ConditionsCOPD, Lung Function Decreased, Alpha 1-Antitrypsin
3 · Its place in the literature

Who cites it

27 citing papers in PubMed, 3 syntheses or guidelines pooled it, 51 citations in OpenAlex.

  1. Pooled it
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  3. Pooled it
  4. Review
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  9. Article
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  11. Article
  12. Definition and Nomenclature of Chronic Obstructive Pulmonary Disease: Time for Its Revision.American journal of respiratory and critical care medicine · 2022
    Article
  13. Self-Amplifying RNA Approach for Protein Replacement Therapy.International journal of molecular sciences · 2022
    Review
  14. Article
  15. Article
  16. Article
  17. Influence ofJournal of the renin-angiotensin-aldosterone system : JRAAS · 2022
    Article
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4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

5 authors at 4 institutions in 5 countries.

Kenneth R ChapmanDepartment of Medicine, University of Toronto, Toronto, ON, Canada.
Joanna Chorostowska-WynimkoDepartment of Genetics and Clinical Immunology, National Institute of Tuberculosis and Lung Diseases, Warsaw, Poland.
A Rembert KoczullaDepartment of Medicine, Pulmonary and Critical Care Medicine, University Medical Center Giessen and Marburg, Philipps-University, Marburg, Germany.
Ilaria FerrarottiCenter for Diagnosis of Inherited Alpha-1 Antitrypsin Deficiency, Department of Internal Medicine and Therapeutics, Pneumology Unit, University of Pavia, Pavia, Italy.
Noel G McElvaneyDepartment of Medicine, Beaumont Hospital, Royal College of Surgeons in Ireland, Dublin, Ireland.
Royal College of Surgeons in Ireland · IEPhilipps University of Marburg · DEUniversity of Pavia · ITUniversity of Toronto · CA

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Alpha 1 antitrypsin deficiency is a hereditary condition characterized by low alpha 1 proteinase inhibitor (also known as alpha 1 antitrypsin [AAT]) serum levels. Reduced levels of AAT allow abnormal degradation of lung tissue, which may ultimately lead to the development of early-onset emphysema. Intravenous infusion of AAT is the only therapeutic option that can be used to maintain levels above the protective threshold. Based on its biochemical efficacy, AAT replacement therapy was approved by the US Food and Drug administration in 1987. However, there remained considerable interest in selecting appropriate outcome measures that could confirm clinical efficacy in a randomized controlled trial setting. Using computed tomography as the primary measure of decline in lung density, the capacity for intravenously administered AAT replacement therapy to slow and modify the course of disease progression was demonstrated for the first time in the Randomized, Placebo-controlled Trial of Augmentation Therapy in Alpha-1 Proteinase Inhibitor Deficiency (RAPID) trial. Following these results, an expert review forum was held at the European Respiratory Society to discuss the findings of the RAPID trial program and how they may change the landscape of alpha 1 antitrypsin emphysema treatment. This review summarizes the results of the RAPID program and the implications for clinical considerations with respect to diagnosis, treatment and management of emphysema due to alpha 1 antitrypsin deficiency.

Indexed as

alpha 1-Antitrypsinalpha 1-Antitrypsin DeficiencyDisease ProgressionHumansLungPulmonary EmphysemaRecovery of FunctionTime FactorsTreatment Outcomealpha 1-AntitrypsinSERPINA1 protein, humanalpha 1 antitrypsin deficiencycomputed tomographyefficacyemphysema

Identifiers

PMID29430176
PMCPMC5797472
OpenAlexW2785382658

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC
Read underepoch 390

Registered trials

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.